Bladder Pain Syndrome MedDRA version: 20.0 Level: LLT Classification code 10071166 Term: Bladder pain syndrome System Organ Class: 100000004857
Conditions
Interventions
Trade Name: BOTOX®
Product Name: BOTOX®
Product Code: 9060X
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: Botulinum Toxin Type A
CAS Number: 93384-43-1
Other descriptive
Sponsors
Centro Hospitalar de São João, EPE
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Female patients aged between 20 and 60 years. - Patients must have pain related to bladder filling, referred to bladder, urethra or perineum for at least 6 months with a score of at least 4 in a 0 (no pain) to 10 (maximum pain) visual analogue scale (VAS). Other urinary complaints such as frequency, urgency or nocturia may be present. - For patients taking oral standard medication for their BPS/IC like PPS, amitriptyline or hydroxyzine must be discontinued at least 15 days prior to screening procedures and a treatment cannot be commenced during the study. - For patients taking intravesical medication like heparinoids, PPS, chondrotin sulfate or DMSO medication must be discontinued at least 3 months prior to screening procedures and a treatment cannot be commenced during the study. - Patients who have received Botulinum toxin type A or resiniferatoxin before, a period of at least 12 months must have elapsed prior to screening procedures. None of these two treatments can be concomitantly commenced during the study. - If the patient is under analgesic medication (which includes paracetamol, non-steroidal anti-inflammatory drugs, gabapentin and pregabalin), she will be allowed at entry. However the dose must remain constant throughout the study duration. If the patient is off analgesic medication no de novo analgesic medication will be allowed during the study. However patients may receive analgesics once they withdraw the study. - Patients must be able and be willing to use clean intermittent catheterization (CIC) at any time after study treatment, if it is determined to be necessary by the investigator. - Anticoagulants, antiplatelet medications, or medications with anticoagulant effects (e.g., warfarin and other coumarinic derivatives, aspirin, clopidogrel, etc) will be prohibited prior to the administration of the study treatment within the following conditions stated in the guideline published by Douketis and colleagues (Douketis et al, 2012): - Vitamin K antagonists will be interrupted for a minimum of 5 days before surgery. - Antiplatelet/antiaggregant drug will be interrupted for a minimum of 7 days before surgery. - These medications could be restarted the day following study treatment. If medically indicated, low molecular weight heparins (e.g. enoxaparin) will be permitted up to 24 hours prior to study drug treatment. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 30 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Pregnant or nursing patients - Patients of childbearing potential that do not use adequate birth control methods (defined in Section 8.2.6) - Patient has history or evidence of any pelvic or urological abnormalities including but not limited to the following: - Current or frequent urinary tract infections (UTI) (> 2 in the last 6 months documented by urine culture) or is taking prophylactic antibiotics to prevent chronic UTI. - History of bladder tumour with or without additional intravesical chemotherapy (mitomycin, BCG). - History of previous pelvic radiotherapy, urinary tract tuberculosis, or exposure to ketamine. - Current or previous non-investigated haematuria (patients with investigated haematuria may enter the study if urological/renal pathology has been ruled out to the satisfaction of the investigator). - Surgery or bladder diseases that may impact bladder function including bladder stones, stress incontinence, endometriosis, uterine prolapse, rectocele, cystocele or any pelvic surgery. - Estimated creatinine clearance rate (eCCr) lower than 30 ml/min, calculated by the Cockcroft-Gault formula: eCCr=((140-Age in years)×Mass (in kilograms))/(72×serum creatinine (in mg/dl))[×0.85 if female] - Cardiac, pulmonary and hematologic conditions or other diseases that may preclude a safe general anaesthesia, in the opinion of the investigator. - Patients at high risk of thrombotic events for whom suspension of antiplatelet or anticoagulant treatment puts patients’ safety at risk. - Patient with a post void residual (PVR) urine volume of > 100 ml at screening. - History of neuromuscular transmission disorders including, but not limited to myasthenia gravis and Eaton Lambert Syndrome - Patients with peripheral motor neuropathic diseases including but not limited to amyotrophic lateral sclerosis. - Allergy to OnabotA or any of the study treatments' componentes. - Administration of OnabotA for other reasons than BPS/IC in the previous 12 weeks.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the efficacy of OnabotA 100 U trigonal injections versus saline in controlling bladder pain, the main symptom referred by patients with BPS/IC, 12 weeks after treatment.;Secondary Objective: - To compare OnabotA versus saline regarding changes in bladder pain intensity at 4 and 8 weeks after treatment. - To compare OnabotA versus saline regarding changes in day-time and night-time urinary frequency 4, 8 and 12 weeks after treatment. - To compare OnabotA versus saline regarding changes in mean urinary volume per void 4, 8 and 12 weeks after treatment. - To compare OnabotA versus saline regarding changes in OLSI scores 4, 8 and 12 weeks after treatment. - To compare OnabotA versus saline regarding changes in quality of life 4, 8 and 12 weeks after treatment. - To compare OnabotA versus saline regarding the benefit reported by patients at 12 weeks after treatment. - To evaluate the safety of OnabotA, for up to 12 weeks after treatment.;Primary end point(s): Absolute variation of pain intensity in pain VAS from visit 1 (screening) to visit 6 (week 12).;Timepoint(s) of evaluation of this end point: From visit 1 (screening) to visit 6 (week 12). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Absolute variation of pain intensity in pain VAS from visit 1 (screening) to visits 4 and 5 (weeks 4 and 8). - Absolute variation of day-time and night-time urinary frequency, measured in the 3D bladder diary from visit 1 (screening) to visits 4, 5 and 6 (weeks 4, 8 and 12). - Absolute variation of mean volume per void, measured in the 3D bladder diary from visit 1 (screening) to visits 4, 5 and 6 (weeks 4, 8 and 12). - Absolute variation in O’Leary-Sant problems and symptoms scores from visit 1 (screening) to visits 4, 5 and 6 (weeks 4, 8 and 12). - Absolute variation of QoL from visit 1 (screening) to visits 4, 5 and 6 (weeks 4, 8 and 12). - Percentage of patients with 50% decrease in pain VAS score at visit 6 (week 12) since visit 1 (screening). - Treatment Benefit Scale score at visit 6 (week 12). - Incidence of adverse events and severe adverse events during the study period. - Incidence of adverse events and severe adverse events whose relationship to the study drug could not be ruled out during the study period.;Timepoint(s) of evaluation of this end point: - Absolute variation of pain intensity in pain VAS: from visit 1 (screening) to visits 4 and 5 (weeks 4 and 8) - day-time and night-time urinary frequency, mean volume per void, O’Leary-Sant score and QoL: from visit 1 (screening) to visits 4, 5 and 6 (weeks 4, 8 and 12) - Percentage of patients with 50% decrease in pain VAS score: at visit 6 (week 12) since visit 1 (screening). - Treatment Benefit Scale score: visit 6 (week 12). - Incidence of adverse events and severe adverse events: during the study period. - Incidence of adverse events and severe adverse events whose relationship to the study drug could not be ruled out during the study period: after the study period. | — |
Countries
Portugal
Contacts
Public ContactProf. Francisco Cruz
Centro Hospitalar de São João, EPE
Outcome results
None listed